Adeno-associated virus compositions for pah gene transfer and methods of use thereof
AssigneeHomology Medicines, Inc.
InventorsAlbert Barnes Seymour, Seemin Seher Ahmed, Jason Boke Wright, Serena Nicole Dollive, Hillard Rubin
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Abstract
Provided herein are adeno-associated virus (AAV) compositions that can express a phenylalanine hydroxylase (PAH) polypeptide in a cell, thereby restoring the PAH gene function. Also provided are methods of use of the AAV compositions, and packaging systems for making the AAV compositions.
Claims5
1-24. (canceled)
A packaging system for preparation of a replication-defective AAV comprising:
- (a) a first nucleotide sequence encoding one or more AAV Rep proteins;
- (b) a second nucleotide sequence encoding an AAV capsid protein;
- and
- (c) a third nucleotide sequence comprising an AAV genome comprising a silently altered PAH coding sequence comprising the nucleotide sequence set forth in SEQ ID NO:
- 25.
A method for treating a subject having a disease or disorder associated with a PAH gene mutation, the method comprising administering to the subject an effective amount of a replication-defective AAV comprising:
- (a) an AAV capsid comprising an AAV capsid protein;
- and
- (b) an AAV genome comprising a silently altered PAH coding sequence comprising the nucleotide sequence set forth in SEQ ID NO:
- 25.
(canceled)
Dependencies are inferred from the claim text.